ISTH 2023 | Unmet needs and emerging treatments in von Willebrand disease

Von Willebrand disease is a very complex disorder and what is known that is that most available current treatment options are kind of effective to stop the bleeding in these patients, but they are not very effective in giving the patients a good quality of life, as demonstrated by the high incidence of depression and anxiety in these patients. So we definitely need more and better treatment options for these patients. So there are new treatment options that are under investigation right now. And in our lab we have explored a few of these options. One of these is a bispecific nanobody that binds concomitantly VWF and albumin and this has been shown in a mouse model of von Willebrand disease type 1 to be effectively able to increase von Willebrand factor antigen levels as well as correcting the bleeding tendency of these mice. There is another molecule that is out there, it’s BT200 and this could be – it’s an aptamer that it can be beneficial in von Willebrand disease type 2B patients that have thrombocytopenia as well as hemophilia A patients. And then it’s interesting to explore the future use of emicizumab in von Willebrand disease patients and this could be useful for… It has been shown that it is successful in von Willebrand disease type 3 patients. But it would be interesting to see if it’s successful in other von Willebrand disease patients and in the lab we have explored this way in a mouse model of von Willebrand disease type 3 and type 2A, and we have to our surprise seen that emicizumab seems to have no effect, at least in our mouse model of von Willebrand disease type 2A. In another study we have shown that synthetic platelets that are able to cooperate with endogenous platelets have been able to ameliorate the bleeding tendency of von Willebrand disease type 3 and type 2B mice with a lot of advantages. So this is another way to explore potential new treatments for von Willebrand disease.